The FDA Approves EMPAVELI, New Treatment Option for C3G and Primary IC-MPGN

In a continued wave of progress for rare kidney disease (RKD) treatments, the FDA has approved the drug EMPAVELI (pegcetacoplan) for use in patients 12 and older with C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). This approval also includes individuals who have experienced disease recurrence after kidney transplant.

C3G and primary IC-MPGN are ultra-rare, progressive diseases that damage the filtering units of the kidneys and can ultimately lead to kidney failure. 

“The approval of EMPAVELI is a historic milestone for people living with C3G and primary IC-MPGN, many of whom are adolescents or young adults,” said Josh Tarnoff, NephCure CEO. “We recognize Apellis’ commitment to these patients and their families, and to the research and innovation that will bring this life-changing treatment into the hands of patients that need it most.”

What the Research Shows

The FDA’s approval of EMPAVELI is based on results from a Phase 3 clinical trial called VALIANT, which looked at how well the treatment worked in people with C3G or primary IC-MPGN.

The study found that patients who received EMPAVELI experienced:

• A 68% reduction in proteinuria, a key sign of disease activity
• Stabilization of kidney function, helping to slow disease progression
• Significant reduction in C3 deposits in the kidneys, with 71% of patients showing no remaining deposits on follow-up testing

What This Means for the RKD Community

With this approval, EMPAVELI becomes an additional option for patients and families navigating life with C3G or primary IC-MPGN. It follows the FDA’s March 2025 approval of FABHALTA for adults with C3G and continues the encouraging trend toward increased treatment options for people with rare kidney diseases.

There are now five FDA-approved treatment options for rare kidney disease:

• EMPAVELI (pegcetacoplan) – Approved for C3G and Primary IC-MPGN
• FABHALTA (iptacopan) – Approved for C3G and IgAN
• FILSPARI (sparsentan) – Approved for IgAN
• TARPEYO (budesonide) – Approved for IgAN
• VANRAFIA (atrasentan) – Approved for IgAN

NephCure remains dedicated to advancing education, support, and advocacy for individuals affected by C3G and other RKDs. The organization encourages patients and families to speak with their healthcare providers to determine whether EMPAVELI or any other available treatment may be appropriate based on individual needs.

To read Apellis’ full press release on EMPAVELI’s approval, click here.

For more information on C3G, IC-MPGN, and available resources, visit NephCure.org.